The Brain Research Through Advancing Innovative Neurotechnologies® Initiative, or The BRAIN Initiative®, has introduced a new era of tool-driven discovery in neuroscience. Over the past decade, this unique effort that brings together 10 NIH Institutes and Centers (ICs), academia, industry, and communities of people living with brain disorders has changed the way we think about the human brain. BRAIN has also changed the way we do neuroscience: shining a light on the value of team science involving many different types of science and scientists.
Yet there is still a lot we don’t know about the brain, and millions of people are counting on research to lead the way. We are driven by the NINDS mission to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease for all people.
Looking forward, the stakes are clear and urgent: one in three people will face a brain disorder in their lifetime. On top of the significant physical and emotional toll is the reality that treating brain injury and disorders costs the United States a staggering $1.5 trillion annually. Meeting this challenge means not only sustaining innovation: it means inventing the future of neuroscience.
A Neuroscience Roadmap for the Next Decade
Neuroscience itself is entering a new phase – one defined not just by discovery, but by the need for integration. Published recently, the Neuroscience Research Roadmap is a call to take stock and look ahead – building on progress and recognizing new scientific trends. The roadmap reflects collaboration across the BRAIN Initiative to identify strategic, technology-driven focus areas that identify new opportunities where BRAIN can have the greatest impact.
The roadmap calls for renewed collaboration among many people and groups committed to the same goal of developing treatments for human brain disorders. Front of mind in this research are complex questions about autonomy, privacy, and potential uses of neurotechnology beyond medicine, requiring careful ethical oversight and input from society as a whole.
Importantly, the roadmap does not replace existing programs but rather provides a strategic framework to connect them and extend their impact. It is organized into four interconnected Innovation Domains: a Brain KnowledgeBase to link diverse datasets into a unified, AI-enabled discovery ecosystem; Precision Molecular Circuit Therapies to enable targeted, mechanistically grounded treatments; Accelerating Human Neuroscience to better connect insights from model systems with studies in people; and NeuroAI to advance both neuroscience and artificial intelligence through shared discovery. Each is a technology-driven area grounded in ethical research, building the talent pipeline, and open science.
The ultimate goal of the Brain Knowledgebase is an AI-enabled research platform that integrates large, complex neuroscience datasets into a unified, interoperable system. By linking existing and new molecular, cellular, circuit, and behavioral data, the knowledgebase should substantially amplify the value of current resources. It also opens new doors to discovery that can better explain how networks of connected brain cells and regions give rise to how we think, remember, feel, learn, and act – ultimately toward the goal of treating human neurological disorders.
Precision Molecular Circuit Therapies builds on tools developed through the BRAIN Initiative and broader neuroscience research. Its aim is to identify and target specific networks of brain cells using genetic methods such as gene delivery to correct dysfunction of brain cells and circuits underlying neurological disorders. We envision key goals of this research as de-risking emerging therapies, capturing knowledge from basic neuroscience research, and integrating ethical and regulatory safeguards early in the research process.
As a result of remarkable progress, new neurotechnologies allow scientists to study human brain activity in detail like never before. A main goal of Accelerating Human Neuroscience is to ensure that lab-based discoveries and tools make their way to testing and ultimately treatments in people – while identifying and addressing ethical questions that arise. Ongoing interactions with regulatory experts and payors and people with lived experience will be necessary for real-world impact of this research.
The human brain is remarkably flexible and efficient (using only the power of a 20-watt lightbulb), but we don’t know how. NeuroAI aims to create a two-way exchange between neuroscience and AI, using newly discovered principles of brain function to improve computer models – while using AI to better understand how “natural” intelligence works in the brain. This work will require new approaches that reflect real-world interactions, along with strong protections for data privacy and responsible data handling.
A Shared Vision
Going forward, the BRAIN Initiative will remain an important research catalyst, working closely with NINDS and other partners across the neuroscience ecosystem, to realize the vision of a Neuroscience Research Roadmap. But success will depend heavily on coordinated collaboration. That means enabling effective and efficient transitions: from discovery to development to implementation among government, academia, industry, philanthropy, international partners, and people living with brain disorders.
Unraveling the complexity of the human brain is truly the challenge of our lifetime. We invite partners across neuroscience to join us in shaping and advancing this shared roadmap, so that together we can turn discovery into health for the millions of people living with brain disorders.
John Ngai, Ph.D.
Director, NIH BRAIN Initiative
Amy Bany Adams, Ph.D.
Acting Director, National Institute of Neurological Disorders and Stroke